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Eculizumab a new drug to treat aHUS

16 April 2019

With funding from Kidney Research UK, Professor Tim Goodship and his team have discovered a new way to treat a rare and life-threatening kidney disease.

Atypical Haemolytic Uraemic Syndrome (aHUS) is a destructive kidney condition that affects the immune system, causing it to attack the patient’s kidney cells. Blood clots form, which block the tiny filters in the kidney, leading to kidney failure.

Until now, the outlook for patients with aHUS has been bleak. There is still no cure and a kidney transplant has not previously been possible as the disease would attack and destroy the new kidney just as it did the patient’s own kidney. The only option for aHUS patients was to begin and remain on an exhausting dialysis routine, which has a huge impact on the quality of their life.

But there is new hope for aHUS patients, thanks to pioneering research at Newcastle University funded by Kidney Research UK.

Professor Tim Goodship
Professor Tim Goodship

A breakthrough drug treatment

Professor Tim Goodship, a researcher at Newcastle University, has made a breakthrough in understanding how to stop the disease aHUS from destroying patients’ kidneys and this ultimately led to the introduction of a pre-existing drug, eculizumab, as a treatment. This drug stops part of the immune system – called the complement- which is overactive in patients with aHUS. This development saves lives because it saves kidneys.

The great news for patients is that this new drug treatment means kidney transplants are now an option. Treating a patient with eculizumab after a kidney transplant can stop the disease from coming back.

The future for patients with aHUS

There has been a step forward towards making this new treatment available to all aHUS patients in the UK. In January 2015, the National Institute for Health and Care Excellence (NICE) recommended eculizumab for use on the NHS. Eculizumab is currently funded by NHS England through interim specialised commissioning arrangements.

aHUS is a very distressing condition that imposes a significant burden both on those with the condition and their carers and families. We are therefore pleased to be able to recommend eculizumab for funding. Sir Andrew Dillon, NICE Chief Executive

We need more vital funding to keep supporting life-saving work like this.

Reviewed April 2019

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